Santa Fe scientists advance in the creation of a drug for neurodegenerative diseases

The human erythropoietintraditionally used to treat anemia, is being re-evaluated by researchers to its possible application in the field of neurodegenerative diseases in the future. The reformulation of this biotherapeutic agent seeks to avoid the adverse effects that it presents when administered to non-anemic patients. The project is still in development, going through stages of research and testing that must be completed to definitively evaluate its usefulness.

The project for this medicine is being led by BioSynaptica SAa start-up company Faculty of Biochemistry and Biological Sciences of the National University of the Litoral (UNL)created by researchers from Conicet. This team aims to innovate in the treatment of neurodegenerative disorders by creating an advanced neurodrug.

In an interview with the media AIRthe scientists Miracles Bürgi and Marcos Oggeropart of the research team, shared details and projections about this contribution to the field of medicine and health.


Milagros Bürgi and Marcos Oggero, along with Gabriela Aparicio and Camila Scorticati.

Santa Fe: advance of a drug for neurodegenerative diseases

In 2013, in the Cell Culture Laboratory of the Faculty of Biochemistry and Biological Sciences of the National University of the Litorala team of researchers and scholars, led by the Dr. Marcos Oggero Eberhardt, started the project. Together with the researcher from Santa Fe Miracles Bürgiwere dedicated to the design, obtaining and production of modified proteins based on human erythropoietin (hEPO).

EPO is a protein produced by humans, known for its main role in the production and maintenance of red blood cells. Due to this function, it is used as a biotherapeutic in patients with chronic anemia. However, It has also been found to play other important roles, including neuroprotection and neuroplasticity.

Miracles Bürgi explained these functions saying: “This protection of neurons from cytotoxic agents or environments and neuroplasticity refers to the ability to reestablish or establish connections between neurons to promote synapsis. The cellular protection function extends to different tissues, as brain, retina and heart, among others.”

“For many years our laboratory worked with human erythropoietin (hEPO), we knew the molecule and its potential to display neuroplasticity and neuroprotection,” he explained. Bürgi. “As there is no effective treatment today, we saw an unmet need and in this way we devised a molecule that could be effective in the treatment of such pathologies,” he added.

From then on, erythropoietin began to be explored as a option for protecting neurons and improving their plasticitywithout the side effects associated with its function of producing red blood cells and other blood elements.

The main objective was to modify this protein to use it in the treatment of neurodegenerative diseases in patients who did not suffer from anemia, thus avoiding adverse side effects such as the excessive increase in the production of red blood cells, the formation of blood clots or other risks that, in In extreme cases, they could be fatal.

To achieve this, the scientists focused on modifying the erythropoietin molecule using a technology called glycoengineering by hyperglycosylation, which is a technology they developed in the laboratory to apply to different proteins.

“What we did was annul the sites of the molecule responsible for triggering this red blood cell production function, but preserving the molecular area responsible for the neurobiological function, which is this capacity for neuroprotection and neuroplasticity,” he explained. Miracles Bürgi.

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The project began in 2013.

What is the current status of the therapeutic candidate?

Currently, the laboratory in Santa Fe is carrying out tests with these molecules in animal models of degenerative retinopathies. Bürgi explained that the decision is based on the perception that the development of this therapeutic candidate for entry into the pharmaceutical market could be expedited by targeting this type of pathologies.

“Especially because for a therapeutic candidate to become a biotherapeutic, it has to go through the clinical phases where patients are treated to evaluate the effectiveness of that molecule,” he indicated.

“So clinical trials in degenerative retinopathy are relatively simpler and shorter because it is easier to recruit patients and see signs of improvement compared to clinical trials for Alzheimer’s or Parkinson’s,” described the researcher from Santa Fe.

For this reason, they are concentrating their efforts on these pathologies to accelerate the progress of the process. In addition, they continue to evaluate the impact on different types of neurodegenerative diseases simultaneously.

Marcos Oggeroan independent researcher at CONICET and professor at UNL, explained that regarding the evidence on neurodegenerative diseases they are waiting to “access their potential treatment given that we have observed that healthy mice treated with the modified hEPO protein showed that brain cells connect in a more elaborate way, which could be very important for brain function and improving interneuronal connection.

However, for the project to continue, the results can be consolidated and a path of more tests is followed, he expressed that “We are looking for investment funds that can finance the development and strengthen the production and analysis phase in animal models of such pathologies.”

And he added: “For this reason, researchers have decided to form BioSynaptica, a scientific and technological-based company that can carry out the steps that are necessary to establish a plan to reach the market. “Market” sounds like a dehumanizing word, but “It is the necessary element for what is applied, in this case a biopharmaceutical, to reach the patient,” he expressed.

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Neurodegenerative diseases are chronic and progressive conditions that affect the nervous system.

Neurodegenerative diseases are chronic and progressive conditions that affect the nervous system.

The steps to follow in the future: how it would be marketed

Right now, they are dedicated to carrying out conceptual tests in animal models and developing the process to produce molecules with the necessary purity and quantity. This will allow them to advance to the preclinical phase and eventually begin clinical trials in the future.

In these future stages, a new evaluation of the toxicity of the molecule in humans will be carried out. Subsequently, we will proceed to the following phases, which will focus on determining the effectiveness of the treatment on the specific pathology that is desired to be treated.

Miracles Bürgi He explained that currently “what we saw with the molecule is that it significantly reduces the death of retinal neurons and protects them from disease or damage, in addition to reducing the formation of neovessels that seriously affect vision. In this sense, it could “Use the molecule preventively in diabetic or premature retinopathies such as those of the newborn to improve or extend the vision of people who suffer from these diseases.”

Regarding the eventual commercialization of the therapeutic candidate, there is still a long way to go, since there are still many validations to be carried out and aspects to be developed.

However, the Santa Fe researcher was able to anticipate that the therapy for degenerative retinopathy would be administered through an intraocular injection, or through multiple injections, depending on the specific treatment.

On the other hand, in the case of neurodegenerative diseases, an approach of administering the drug by injection would be considered, that is, through one or several layers of the skin or mucous membranes. The form of administration would vary depending on the type of specific pathology.

Even so, Bürgi He emphasized once again the importance of remembering that this entire process still has a long way to go.

It still needs to be tested and proven to have an effect. Then, think about the dose and the form of treatment“he concluded Miracles Bürgi.

frames Oggero He also highlighted again: “a lot of anxiety is generated in people affected by pathologies, something that is understood very deeply from feelings.”

And he added: “For this reason, we must explain that we are still in a testing phase and that at the moment the pathology that we have addressed the most is degenerative retinopathy, which is a pathology that involves neurodegenerative diseases and for which we hope to continue advancing with the good results.” results we have had.

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